Komiyama O, Hirata A, Handa J
Ad Hoc Committee on Statistics, Japan Pharmaceutical and Manufacturers Association, Tokyo, Japan.
Nihon Yakurigaku Zasshi. 2000 Jul;116(1):12-7. doi: 10.1254/fpj.116.12.
When pharmaceutical scientists describe characteristics of a drug or when they decide whether it is appropriate to initiate clinical trials to determine the drug's effects in humans, their inferences are frequently grounded in information drawn from non-clinical studies. Therefore, certain and highly objective information is required. By introducing the concept of design of experiments to control some nuisance factors and performing confirmatory studies based on sample size estimation, trustworthy information can be efficiently obtained. This paper does not demand that researchers conduct an additional confirmatory study in a series of studies conducted so far. This is a reconsideration how a series of studies should be carried forward. Statistics ought to contribute much more not only to estimation or hypothetical tests after data are collected, but also to methodology of preliminary experiments and planning of studies. Cooperation with statisticians from an early stage of the studies is all the more helpful in non-clinical studies, in which, in a sense, "perfect" experiments can be conducted more than in clinical studies.
当药物科学家描述一种药物的特性，或者决定是否适合开展临床试验以确定药物对人体的作用时，他们的推断常常基于从非临床研究中获取的信息。因此，需要确定且高度客观的信息。通过引入实验设计的概念来控制一些干扰因素，并基于样本量估计进行验证性研究，可以有效地获得可靠信息。本文并不要求研究人员在迄今为止进行的一系列研究中再开展一项额外的验证性研究。这是对一系列研究应如何推进的重新思考。统计学不仅应该在数据收集后对估计或假设检验做出更大贡献，还应该对初步实验的方法和研究规划做出更大贡献。在研究的早期阶段就与统计学家合作，在非临床研究中更有帮助，因为在某种意义上，非临床研究比临床研究更能进行“完美”的实验。
